FDA Approves First Drug for a Form of Batten Disease

On Thursday, April 27th, the FDA announced the approval of Brineura™ (cerliponase alfa). Brineura, manufactured by BioMarin International Ltd., is an infusion indicated for the treatment of symptomatic children ages three and older suffering from late infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease, which is a form of Batten disease.

What is CLN2?

CLN2 disease is an inherited and extremely rare and progressive fatal brain condition that primarily affects the nervous system. The condition affects less than one in one million U.S. residents, and many patients with the disease go undiagnosed. In a recent press release, FDA states that signs and symptoms of the late infantile form of the disease typically begin between ages two and four, and include:

Language delay

Recurrent seizures (epilepsy)

Difficulty coordinating movements (ataxia)

Muscle twitches (myoclonus)

Vision loss

Children with the condition often have trouble with essential motor skills, such as sitting and walking. Because of this, many of these children require the use of a wheelchair by late childhood, and rarely survive past their teen years.

FDA Approves 1st CLN2 Treatment

Brineura was approved by the FDA last week, marking a huge step forward for patients with CLN2 and their families. Brineura is an enzyme replacement therapy. The active ingredient in the product is cerliponase alfa, which “is a recombinant form of human TPP1, the enzyme deficient in patients with CLN2 disease.”

“Brineura is administered into the cerebrospinal fluid (CSF) by infusion via a specific surgically implanted reservoir and catheter in the head (intraventricular access device).” To reduce the risk of infections, the product must be administered to patients under sterile conditions. FDA states that a healthcare professional knowledgeable in intraventricular administration should manage the treatment.

For pediatric patients ages three and older, the recommended dose of Brineura is a 300mg intraventricular infusion, followed by an infusion of electrolytes. It is recommended that children receive this dose on a bi-weekly basis.

Evaluating Safety & Efficacy

BioMarin established the efficacy of Bruneura “in a non-randomized, single-arm dose escalation clinical study in 22 symptomatic pediatric patients with CLN2 disease and compared to 42 untreated patients with CLN2 disease from a natural history cohort (an independent historical control group) who were at least 3 years old and had motor or language symptoms.” FDA notes that age, baseline walking ability, and genotype were taking into account. According to a press release from BioMarin, the product “was shown to slow the loss of ambulation in symptomatic pediatric patients 3 years of age and older with CLN2 disease.”

FDA states that “the safety of Brineura was evaluated in 24 patients with CLN2 disease aged 3 to 8 years who received at least one dose of Brineura in clinical studies. The safety and effectiveness of Brineura have not been established in patients less than 3 years of age.”

Side Effects of Brineura

The most common adverse reactions experienced when using Bruneura include:

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